Gaucher Disease
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-08-25EMA Authorised: Vpriv (velaglucerase alfa) — Takeda Pharmaceuticals International AG Ireland Branch
The EMA authorises Vpriv (velaglucerase alfa) for Gaucher disease under accelerated assessment.
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EMA approval · 2026-07-27EMA Authorised: Cerdelga (Eliglustat (tartrate)) — Sanofi B.V.
Cerdelga (Eliglustat tartrate) has been authorised by EMA for Gaucher disease.
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EMA approval · 2026-07-09EMA Authorised: Zavesca (miglustat) — Advanz Pharma Limited
Authorization granted for Zavesca (miglustat) to Advanz Pharma Limited for Gaucher disease and Niemann-Pick diseases in the EU.
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Genetic Studies of Lysosomal Storage Disorders
A genetic study of lysosomal storage disorders, including Gaucher disease and potentially Parkinson disease, is currently enrolling by invit…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) [Phase 1, Phase 2]
Clinical trial PROCEED is a Phase 1/2 study evaluating PR001 (LY3884961) for peripheral manifestations of Gaucher disease, and the trial is …
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CLINICAL_TRIALS research · 2026-04-14Clinical Trial Update: International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
An international Gaucher disease registry and pregnancy sub-registry sponsored by Genzyme (Sanofi) is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
A clinical trial exploring extracellular vesicles as potential biomarkers and a therapeutic target for Gaucher disease is currently recruiti…
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CLINICAL_TRIALS trial_result · 2026-04-06Clinical Trial Update: Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
NHGRI-sponsored clinical trial evaluating positron emission tomography imaging in people with Gaucher mutations (including those with Parkin…
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CLINICAL_TRIALS trial_result · 2026-04-03Clinical Trial Update: High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3) [NA]
The trial evaluating high-dose ambroxol for pediatric Gaucher disease type 3 has been terminated.
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
A survey is being conducted to evaluate how participants, caregivers, and nurses use and understand educational materials for Velaglucerase …
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CLINICAL_TRIALS other · 2026-03-27Clinical Trial Update: A Study of Home vs Hospital Treatment in People With Fabry, Gaucher or Hunter Conditions in Mexico
ClinicalTrials.gov update on a study comparing home vs hospital treatment for Fabry disease, Gaucher disease, and Hunter syndrome in Mexico,…
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CLINICAL_TRIALS trial_result · 2026-02-19Clinical Trial Update: PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
Clinical trial NCT05641103 (PREDIGA 2), an Educational and Diagnostic Project for Gaucher disease and acid sphingomyelinase deficiency, has …
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
Clinical trial update for GammaGA examining how common acid sphingomyelinase deficiency (ASMD) and Gaucher disease are in patients with mono…
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EMA approval · 2026-01-28EMA Authorised: Zavesca (miglustat) — Janssen Cilag International NV
EMA authorised Zavesca (miglustat) for Gaucher disease and Niemann-Pick diseases; Janssen Cilag International NV is the marketing authorisat…
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EMA approval · 2026-01-28EMA Authorised: Vpriv (velaglucerase alfa) — Takeda Pharmaceuticals International AG Ireland Branch
The EMA authorised Vpriv (velaglucerase alfa) for Gaucher disease, with accelerated assessment.
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CLINICAL_TRIALS research · 2026-01-14Clinical Trial Update: Institutional Registry of Rare Diseases
An institutional registry for rare diseases sponsored by Hospital Italiano de Buenos Aires is currently recruiting. The registry includes am…
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FDA approval · 2026-01-12FDA Approved: CEREZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Cerezyme (imiglucerase) from Genzyme Corporation, resulting in an updated labeling for the produ…
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FDA label_change · 2025-12-22FDA Approved: ELELYSO — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
FDA approved a supplemental application for ELELYSO (taliglucerase alfa) submitted by Pfizer Laboratories Div Pfizer Inc, resulting in chang…
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EMA approval · 2025-12-19EMA Authorised: Yargesa (miglustat) — Piramal Critical Care B.V.
The European Medicines Agency authorised Yargesa (miglustat) for Gaucher disease, with Piramal Critical Care B.V. as the marketing authorisa…
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EMA approval · 2025-12-01EMA Authorised: Cerezyme (imiglucerase) — Sanofi B.V.
EMA authorised Cerezyme (imiglucerase) for Gaucher disease, with Sanofi B.V. listed as the marketing authorization holder.
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CLINICAL_TRIALS research · 2025-10-09Clinical Trial Update: Gaucher Disease Outcome Survey (GOS)
The Gaucher Disease Outcome Survey (GOS) is a recruiting clinical trial sponsored by Shire to collect data on Gaucher disease.
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen
A non-interventional national pediatric study sponsored by Sanofi investigating unexplained enlarged spleen in patients with Gaucher disease…
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CLINICAL_TRIALS research · 2025-09-22Clinical Trial Update: A Long-term Follow-up Study of Gaucher Disease
This is a long-term follow-up clinical study on Gaucher disease that is currently recruiting at Duke University.
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CLINICAL_TRIALS trial_result · 2025-09-15Clinical Trial Update: Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ [Phase 4]
A Phase 4 study assessing the efficacy and safety of imiglucerase in Chinese patients with Gaucher disease type III has been completed.
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CLINICAL_TRIALS research · 2025-09-12Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…